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Journey to the Center of the Cell: Tracing the Path of AAV Transduction
Open accessAbstract
As adeno-associated virus (AAV)-based gene therapies are being increasingly approved for use in humans, it is important that we understand vector-host interactions in detail. With the advances in genome-wide genetic screening tools, a clear picture of AAV-host interactions is beginning to emerge. Understanding these interactions can provide insights into the viral life cycle. Accordingly, novel strategies to circumvent the current limitations of AAV-based vectors may be explored. Here, we summarize our current understanding of the various stages in the journey of the vector from the cell surface to the nucleus and contextualize the roles of recently identified host factors.
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“After endosomal escape, rAAV particles either enter the nucleus for transgene expression, or become ubiquitylated then degraded by proteasome (84).”
From Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies (2021) · cites this paper as [84]
In the citing paper
“However, endosomal acidification is essential for rAAV escape (84), and hydroxychloroquine increases endosomal and lysosomal pH (60), this effect may not be replicated or consistent with systemic application.”
From Immunomodulation in Administration of RAAV: Preclinical and Clinical Adjuvant Pharmacotherapies (2021) · cites this paper as [84]
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“Adeno-associated virus (AAV) is an attractive vector in human gene therapy because it is a nonpathogenic parvovirus.(32) Double-strand (ds) AAV DNA, generated from replication of its single-strand DNA, enables CRISPR FISHer targeting (Fig.”
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“On the other hand, the efforts to identify receptors for novel capsids have significant theoretical meanings, as AAV transduction is a complicated and yet-to-be-elucidated process that involves multiple steps that can affect transgene expression [89].”
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“Recent study elucidated that AAVR interacting VR-I also participates in AAV9 galactose binding and might have been selected during evolution to ensure the virus internalization;(15) this is in line with the role of AAVR facilitating intracellular trafficking of the virus.(9,28)”
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“Whether the difference in VGCN reported in short- versus long-term studies reflects non-functional vector genomes that were not taken up by cells and are cleared with time, stabilization of only an initial fraction of the vector genomes in the nucleus, or turnover of some of the targeted cells remains to be determined…”
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“75,87 This phenomenon has been hypothesized to result from species-specific intracellular processing mechanisms 88 or epigenetic regulation of the AAV genome unique to each species.”
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“Beyond Ad5, other commonly used types of viruses are the adeno-associated (AAV), a class of non-pathological viruses characterized by a broad tropism toward different cells thanks to their recognition of glycans, glycoconjugates, or sialic acid [97].”
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“During infection, a conformational change in the capsid is necessary, resulting in the protrusion of the N-termini of VP1u, leading to endosomal escape via PLA2 activity, and of the VP2u out of the intact capsid, routing the capsid to the nucleus by interacting with the NLSs [37,80].”
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“Other viruses instead—AAV8 included—need to survive and accomplish endosomal escape intact in order to complete the infection [38,39].”
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“85 Disease processes that affect intracellular traf fi cking can block the ef fi ciency of AAV transduction.”
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Hippocampal CA2 neurons disproportionately express AAV-delivered genetic cargoPreprint
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“This is followed by viral assembly and release (Figure 2) [18]. followed by rep protein synthesis (11), viral assembly, and release (12) (Created in BioRender.com).”
Chai, S., Kim, Y., Galivo, F., Dorrell, C., Wakefield, L., Posey, J., Ackermann, A. M., Kaestner, K. H., Hebrok, M., & Grompe, M. (2022) · Human Gene Therapy · doi:10.1089/hum.2021.219 · Cited by 3
CRISPR screen for rAAV production implicates genes associated with infectionPreprint
O'Driscoll, E., Arora, S., Lang, J. F., Davidson, B. L., & Shalem, O. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.09.17.613356 · Cited by 3
O'Driscoll, E., Arora, S., Lang, J. F., Davidson, B. L., & Shalem, O. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101408 · Cited by 5
Dogbey, D. M., Naran, K., & Barth, S. (2025) · Advances in Cell and Gene Therapy · doi:10.1155/acg2/8881294 · Cited by 2
“Herein, we demonstrated a shift from clathrin and caveolin-mediated cell entry by wild type AAVs [43] to receptor-dependent transduction by the engineered vectors.”
The Cold Case Files of rAAV Capsid Influence on Transduction: New Leads
Powell, S. (2025) · Viruses · doi:10.3390/v17111476 · Cited by 2
“An overview of rAAV transduction. rAAV transduction steps have been thoroughly reviewed elsewhere [18] but is briefly depicted above in numerical order. rAAV binds to cellular attachment factors as well as primary and secondary receptors (step 1) which enable cellular internalization in step 2.…”
Epilepsy Treatment through Genetic Intervention of GABAergic Interneurons: Promises and Challenges
Podgorny, O. V., Petukhova, E. O., & Belousov, V. V. (2025) · ACS Chemical Neuroscience · doi:10.1021/acschemneuro.5c00747 · Cited by 2
Synthetic serum markers enable noninvasive monitoring of gene expression in primate brains
Lee, S., Romac, M. D., Watanabe, S., Chernov, M. M., Li, H., Raisley, E. K., Rothenhoefer, K. M., Dahlquist, Z., Szablowski, J. O., & Costa, V. D. (2026) · Neuron · doi:10.1016/j.neuron.2026.01.003 · Cited by 2
Tissue‐specific gene delivery approaches
Nasr, S. S., Cheema, Y., Stern, A., Tabah, O., Poore, S., & Duncan, G. A. (2026) · Bioengineering & Translational Medicine · doi:10.1002/btm2.70125 · Cited by 3
Munir, M., Butcher, N. J., Werder, R. B., Ranganathan, S., Burow, R., Venables, A., & Kaminskas, L. M. (2026) · Nanomedicine · doi:10.1080/17435889.2026.2640157 · Cited by 2
“Due to their smaller size, AAV then have a better capacity to enter the nucleus without requiring initial capsid disintegration [84].”
Hyperthermia Enhances Adeno-Associated Virus Vector Transduction Efficiency in Melanoma Cells
Bieńkowska, A., Stelmaszczyk‐Emmel, A., Demkow, U. A., & Małecki, M. (2023) · Current Issues in Molecular Biology · doi:10.3390/cimb45100537 · Cited by 2
“Interestingly, HSP70 supports clathrin-dependent endocytosis [62], which is known to be one of the ways that rAAV enters cells [63].”
BackgroundGene therapy targeting the blood-brain barrier
Körbelin, J., Pereira, A. A., & Schwaninger, M. (2024) · Vitamins and hormones · doi:10.1016/bs.vh.2024.03.001 · Cited by 2
Volf, N., Vuerich, R., Colliva, A., Volpe, M. C., Marengon, M., Zentilin, L., Giacca, M., Ring, N., Vodret, S., Braga, L., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.08.014 · Cited by 2
“…gene therapy, HTS has been used to trace the path of AAV vectors inside mammalian cells and discover major determinants of AAV transduction 15,16 This is the fi rst HTS aiming at identifying FDA-approved drugs able to increase the ef fi ciency of transduction using AAV vectors, specifically…”
Kasden, C. M., Corzo, M. P., Greer, C. D., Singh, T., Reyes, R., Castillo, A., Arany, Z. P., Kozyak, B. W., & Stedman, H. H. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.07.013 · Cited by 2
Artificial miRNAs are potential gene therapy tools, especially for incurable monogenic disordersPreprint
Dowaidar, M. (2021) · OSF Preprints (OSF Preprints) · doi:10.31219/osf.io/d5rnm · Cited by 2
Fustinoni, C., Liu, X., Chen, Z., Cawood, R., Valenti, W., Patrício, M. I., & Su, W. (2025) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2025.101492 · Cited by 1
“13,15”
Ren, H., Nie, J., Song, Z., Mo, W., Yang, Y., & Bai, Z. (2026) · Synthetic and Systems Biotechnology · doi:10.1016/j.synbio.2025.12.018 · Cited by 1
“As biosynthesis of AAV is a complex process, we speculate that additional steps such as genome processing, genome encapsidation, and virion release may affect AAV production as well [16,42,43].”
New Approaches to Cardiac Gene Therapy Delivery
Veera, S., Lin, N., Mavropoulos, S. A., & Ishikawa, K. (2026) · Canadian Journal of Cardiology · doi:10.1016/j.cjca.2026.02.031 · Cited by 1
Becker, R., Choudhury, P., Oti, M., Fouani, Y., Strobel, B., Ketterer, S., Rumpel, S., Park, J., Kreuz, S., Maier, U., et al. (2026) · iScience · doi:10.1016/j.isci.2026.115469 · Cited by 1
“62,63 Indeed, a study comparing different plasmid-and lentivirus-based MPRA approaches found that enhancer activity can be influenced by relative positioning of the enhancer but also by other screening backbone design differences.”
Deep-sequencing-guided library screening and profiling of AAV capsids in the primate retina
Fouani, Y., Blazevic, D., Oti, M., Becker, R., Strobel, B., Schönberger, T., Stephan, M., Wichert, S., Reichel, F. F., Seitz, I. P., et al. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201764 · Cited by 1
TBC1D23–AAVR interaction drives endosome-to-TGN trafficking required for rAAV transduction
Zhang, X., Habib, A., McFarlin, S., Richart, D., Park, S. Y., Cheng, F., Wang, J., Yan, Z., & Qiu, J. (2026) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2605086123 · Cited by 2
Recent advancements in improving cross-species applicability of bioengineered AAV capsids
Pan, H., Li, N., Qi, J., & Chai, R. (2026) · Gene Therapy · doi:10.1038/s41434-026-00628-y · Cited by 1
“Through cross-species research, we can also identify receptors for novel capsids [83, 84], which helps elucidate the mechanism of AAV transduction and transgene expression [85].”
The predawn dilemma in adeno-associated virus-based gene therapies for hereditary deafness
Zhou, J. (2025) · American Journal of Stem Cells · doi:10.62347/fesn5062 · Cited by 1
“Once released, single-stranded DNA is converted to double-stranded DNA, resulting in the expression of the recombinant transgene [37-39].”
Adeno-associated viruses escort nanomaterials to specific cells and tissuesPreprint
Nagao, K., Paniagua, E. V., Lei, K., Beckham, J. L., Worthington, P., Manthey, M., Ye, M., Koehler, F., Kim, Y. J., Malkin, E., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.04.04.647267 · Cited by 2
Mole, A. J., Sander, C. F., Parmar, A., Williams, A. J., Azzouz, M., Hautbergue, G. M., Shaw, P. J., Ferraiuolo, L., & Mead, R. J. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.07.01.662642 · Cited by 1
Targeting the nociceptive somatosensory system with AAV9 and AAV2retro viral vectorsPreprint
Skorput, A. G. J., Gore, R., Schorn, R. E., Riedl, M. S., de Velasco, E. M. F., Hadlich, B., Kitto, K. F., Fairbanks, C. A., & Vulchanova, L. (2021) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2021.05.25.445559
Becker, R., Choudhury, P., Oti, M., Fouani, Y., Strobel, B., Ketterer, S., Rumpel, S., Park, J., Kreuz, S., Maier, U., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.06.04.657008
“It appears possible that this position-sensitive enhancer activity is related to aspects of AAV genome biology, for example the oligomerization of episomal AAV genomes into concatemers (Yang et al. 1999; Dhungel et al. 2021).”
A packaging-regulated adenoviral vector for rAAV production
Lu, D., Li, R., Liang, Q., Xiao, Z., & Liu, Q. (2025) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2025.152326
Increased in vivo transduction of AAV-9 cargo in Alport podocytes
Williams, E. G., Fresquet, M., Bolás, G., Kaseda, S., Goncalves, K. A., Steinsapir, A., Adamson, A. D., Sherwood, D. R., & Lennon, R. (2025) · Wellcome Open Research · doi:10.12688/wellcomeopenres.24212.1
“To further enhance transduction efficiency, and cell specificity additional strategies could be employed, including tailoring the AAV by customizing cell-specific promoters, optimizing transgene designs, and engineering capsid variants to achieve specific tissue tropism 24 .”
An optimised method for generation of murine CAR-T cells by CRISPR/Cas9Preprint
Jackson, T. J., Himsworth, C., Munnings-Tomes, S., Alam, F., Brezovjakova, H., Donovan, L., Erbe, A. K., Sondel, P. M., Chesler, L., & Anderson, J. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.11.29.689298
Leysen, M., Peredo, N., Pavie, B., Moeyaert, B., & Henckaerts, E. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms27010236
“Multiple cytoplasmic trafficking routes have been proposed, involving early, late, and recycling endosomes [11,13,17,18], as well as cytoskeletal elements such as microtubules and dynein [17,19–22].”
Influential
Metadata
- Authors
- Dhungel, B. P., Bailey, C. G., & Rasko, J. E. J.
- Affiliations
- The University of Sydney (AU)
- Corresponding author
- Rasko, John E.J.
- Venue
- Trends in Molecular Medicine
- Publisher
- Elsevier BV
- Published
- 2021-02
- Volume
- 27
- Issue
- 2
- Pages
- 172-184
- ISSN
- 1471-4914 (ISSN-L)
- OpenAlex
- W3092811898
- Cited by
- 122
- References
- 139
- Citations by year
- 2026: 32 · 2025: 29 · 2024: 21 · 2023: 17 · 2022: 9 · 2021: 14
- FWCI
- 1.43
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Trends in Molecular Medicine (publishedVersion)
- PubMed (publishedVersion)
- Funders
- NSW Health
- National Health and Medical Research Council
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
- Keywords
- adeno-associated virus, adeno-associated virus vectors, gene therapy, vector-host interactions, viral life cycle, host factors, nuclear translocation, viral transduction, genome-wide screen
- MeSH terms
- Host Microbial Interactions, Dependovirus, Animals, Biological Transport, Genetic Vectors, Humans, Transduction, Genetic, Genetic Therapy, Gene Transfer Techniques, Cell Tracking
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